After amyloid beta, the second big target in Alzheimer’s disease centers on toxic accumulations of tau, possibly working hand-in-hand with a-beta to fry people’s memories. And today Ionis $IONS says that it’s picking up a $10 million milestone from its partners at Biogen to begin an early-stage study that will set out to try and determine if its antisense tech can make a difference in disease progression.
Researchers plan to enlist 44 patients with mild Alzheimer’s in the study of IONS-MAPTrx to make sure it’s safe and look for initial signs of drug activity over three months. Biogen — which has an ambitious late-stage effort underway in amyloid beta with aducanumab — has an option on the work.
“In contrast to amyloid plaques that may begin to deposit in the brain for up to 20 years before the onset of AD, tau deposits are spatially and temporally associated with the brain regions where atrophy occurs and neurocognitive deficits originate. IONIS-MAPTRx is designed to reduce the production of all of the many forms of tau in all regions of the brain,” said C. Frank Bennett, senior vice president of research at Ionis. If it is active, Ionis plans to pursue a two-track approach to tauopathies, with a separate program planned for frontotemporal dementia.
It’s a high-risk effort. Alzheimer’s has defeated everything thrown at it in Phase III over the past 15 years, including the recent 5HT6 effort at Axovant which flat failed to bend the curve on this disease.
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